Hechmati Guy, Hauber A Brett, Arellano Jorge, Mohamed Ateesha F, Qian Yi, Gatta Francesca, Haynes Ian, Bahl Amit, von Moos Roger, Body Jean-Jacques
Global Health Economics, Amgen (Europe) GmbH, Dammstrasse 23, Zug, 6301, Switzerland,
Support Care Cancer. 2015 Jan;23(1):21-8. doi: 10.1007/s00520-014-2309-x. Epub 2014 Jun 18.
The aim of this study was to assess patients' preferences for efficacy, safety, and mode of administration in relation to available bone-targeted agents (BTA) for the prevention of skeletal-related events (SREs) associated with bone metastases in Europe.
Adults in France (n = 159), Germany (n = 166), and the United Kingdom (UK; n = 159) with a self-reported physician diagnosis of bone metastases secondary to a solid tumour completed an online discrete- choice experiment survey of ten questions, choosing between pairs of hypothetical BTA profiles. Profiles were defined by five treatment attributes: delay of first SRE, delay of worsening of pain, annual risk of osteonecrosis of the jaw (ONJ), annual risk of renal impairment, and mode of administration. Profiles were generated using an experimental design with known statistical properties. A main-effects random parameters logit (RPL) model was applied to relate participants' choices to the characteristics of the BTA profiles.
The most important treatment attributes for patients across all three countries were time until first SRE, annual risk of renal complications and time until pain worsening. For these attributes, better levels of outcomes were significantly preferred to worse levels (p < 0.05). A 120-minutes infusion every 4 weeks was the least preferred mode of administration. Risk of ONJ was judged by patients in the UK and Germany to be the least important attribute.
Patients consider delaying SREs, avoiding renal impairment and delaying pain worsening as the most important goals to consider when selecting treatment to prevent the bone complications commonly associated with bone metastases.
本研究旨在评估欧洲患者对于现有骨靶向药物（BTA）在预防与骨转移相关的骨相关事件（SRE）方面在疗效、安全性和给药方式上的偏好。
法国（n = 159）、德国（n = 166）和英国（n = 159）自我报告经医生诊断为实体瘤继发骨转移的成年人完成了一项包含十个问题的在线离散选择实验调查，在成对的假设BTA简介之间进行选择。简介由五个治疗属性定义：首次SRE的延迟、疼痛加重的延迟、颌骨坏死（ONJ）的年度风险、肾功能损害的年度风险以及给药方式。简介使用具有已知统计特性的实验设计生成。应用主效应随机参数logit（RPL）模型将参与者的选择与BTA简介的特征相关联。
所有三个国家的患者最重要的治疗属性是直至首次SRE的时间、肾脏并发症的年度风险以及直至疼痛加重的时间。对于这些属性，更好的结果水平明显比更差的水平更受青睐（p < 0.05）。每4周一次120分钟的输注是最不受青睐的给药方式。英国和德国的患者认为ONJ风险是最不重要的属性。
患者在选择预防通常与骨转移相关的骨并发症的治疗时，将延迟SREs、避免肾功能损害和延迟疼痛加重视为最重要的目标。
